Treatments for MDS

Our team at Mount Sinai has been in the forefront of researching Myelodysplastic Syndrome (MDS) and developing treatments for this disease. One of our physicians, Dr. Lewis R. Silverman, was instrumental in gaining FDA approval of azacitidine or Vidaza®, the first drug to treat MDS in the U.S. Our team of physicians, researchers, nurses, and social workers are committed to helping patients with MDS live with the best quality of life possible.

Since we know that MDS shows up differently in every patient, it is important to have a range of treatments to draw upon. And because every patient responds differently, we individualize the therapy for each patient. As we develop a diagnosis, we look at the patient’s genomic (mutation) analysis and use that information to tailor the specific therapy. Understanding the mutations helps us identify patients who are more likely to respond to a given therapy. We try to develop treatment strategies that deal with the disease in its current state, but also look at the long-term to improve outcomes for patients.

We start with the diagnosis, which is formed by looking at the results of blood, bone marrow, and genetic testing. The combination of these factors allows us to classify patients in groups, which are established by independent medical associations. These groupings help us predict how the disease will behave in a specific individual.

Treatments may be determined by whether the patient has lower risk disease or higher risk disease. People who are in a lower risk category may have a form of MDS in which it is stalled for several years, and not require treatment. And there are patients with higher risk disease who require treatment much sooner, or even immediately. Treatments along this continuum of care, from lower risk to high risk patients, may include:

  • Observation: Watching and monitoring the progress of the disease is appropriate for patients with low risk disease whose blood counts do not yet require transfusions and whose overall disease can remain stable for months or even years.
  • Supportive care: Transfusions and antibiotics can boost red blood cell and platelet counts, while IV antibiotics can reduce the risk of serious infections. Treatment will vary, depending on which blood cell counts are low.
  • Drug therapies: Dr. Lewis R. Silverman pioneered the use of azacitidine, which is the front line of therapy of treating higher risk MDS around the world. It helps the cells within the bone marrow to regain the ability to produce blood cells normally, eliminating the need for transfusions. However, not every patient responds to this treatment, and it loses its effectiveness over time. So the team at Mount Sinai continually looks for other drugs, used alone or in combination with other drugs, to give each patient their best outcome. For lower risk patients, there are other drugs available that can help reduce or eliminate the need for red cell transfusions.
  • Clinical trials: As part of this ongoing search for more effective treatment, the team at Mount Sinai offers clinical trials for investigational therapies. In tandem with researchers at the Human Immune Monitoring Core and Experimental Therapeutics Institute at the Icahn School of Medicine at Mount Sinai and the Tisch Cancer Institute, the MDS team has a robust program for cutting edge therapies.
  • Stem cell transplants: A stem cell transplant essentially uses blood stem cells from another individual to replace the patient’s own bone marrow and “reboots” the ability of the bone marrow to produce blood cells. The newly donated stem cells also alter the patient’s own immune system and the donated immune cells attack and destroy the patient’s MDS cells. Not every patient can tolerate this procedure; however, if successful, it can actually cure MDS and put it in remission.

Each patient’s journey is different, and at each step, we provide options and strategies to combat the disease and enjoy a better quality of life. However, our medical expertise is just part of the total picture. Our nurse practitioners, nurses, social workers, counselors, and other staff not only help patients and their families live with the disease, but live better. At Mount Sinai, you have a full team working with you for better health.